Lifeline for hunter syndrome patients: continued access to Brain-Targeting therapy

NCT ID NCT05795361

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This program offers continued treatment with idursulfase-IT (TAK-609), given directly into the spinal fluid, plus standard intravenous Elaprase, for children and adults with Hunter syndrome who have cognitive impairment. It is only open to people who completed earlier studies of this combination. The goal is to maintain any benefits they experienced and keep the disease under control.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

idursulfase-IT (TAK-609) given into the spinal fluid, plus intravenous Elaprase

What this could lead to

If successful, this program could provide ongoing disease control and cognitive benefit for people with Hunter syndrome who have cognitive impairment.

What could go wrong

This is an open-access program, not a new trial, so it does not test whether the treatment works. Risks include side effects from the spinal injections or the implanted delivery device.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

mucopolysaccharidosis type 2

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    AVAILABLE

    Chicago, Illinois, 60611, United States

    Contact

    Contact Email: •••••@•••••

  • Board of Regents of the University of Nebraska

    AVAILABLE

    Omaha, Nebraska, 68198-5450, United States

    Contact Email: •••••@•••••

    Contact

  • Division of Medical Genetics, University of Utah

    AVAILABLE

    Salt Lake City, Utah, 84112, United States

    Contact

    Contact Email: •••••@•••••

  • H.C.U. de Valladolid

    AVAILABLE

    Valladolid, 47003, Mexico

    Contact

    Contact Email: •••••@•••••

  • Hospital Universitario Reina Sofia

    AVAILABLE

    Córdoba, 14004, Spain

    Contact

    Contact Email: •••••@•••••

  • Instituto Nacional de Pediatria

    AVAILABLE

    Coyoacán, Mexico City, 4530, Mexico

    Contact

    Contact Email: •••••@•••••

  • Jackson Memorial Hospital University of Miami

    AVAILABLE

    Miami, Florida, 33101, United States

    Contact Email: •••••@•••••

    Contact

  • Joseph M. Sanzari Children's Hospital

    AVAILABLE

    Hackensack, New Jersey, 07601, United States

    Contact

    Contact Email: •••••@•••••

  • NYU Langone Medical Center

    AVAILABLE

    New York, New York, 10017, United States

    Contact

    Contact Email: •••••@•••••

  • Phoenix Childrens Hospital

    AVAILABLE

    Phoenix, Arizona, 85015, United States

    Contact

    Contact Email: •••••@•••••

  • Queensland Childrens Hospital

    AVAILABLE

    South Brisbane, Queensland, QLD 4101, Australia

    Contact

    Contact Email: •••••@•••••

  • Randall Children's Hospital at Legacy Emanuel

    AVAILABLE

    Portland, Oregon, 97227, United States

    Contact

    Contact Email: •••••@•••••

  • Royal Manchester Children's Hospital - PPDS

    AVAILABLE

    Manchester, M13 9WL, United Kingdom

    Contact

    Contact Email: •••••@•••••

  • Seattle Children's Hospital - PIN

    AVAILABLE

    Seattle, Washington, 98105, United States

    Contact

    Contact Email: •••••@•••••

  • The Children's Hospital of Philadelphia

    AVAILABLE

    Philadelphia, Pennsylvania, 19104, United States

    Contact

    Contact Email: •••••@•••••

  • The Cleveland Clinic Foundation

    AVAILABLE

    Cleveland, Ohio, 44195, United States

    Contact

    Contact Email: •••••@•••••

  • The Nemours Foundation

    AVAILABLE

    Wilmington, Delaware, 19803, United States

    Contact

    Contact Email: •••••@•••••

  • The Regents of the University of California

    AVAILABLE

    Oakland, California, 94609, United States

    Contact

    Contact Email: •••••@•••••

  • The University of North Carolina

    AVAILABLE

    Chapel Hill, North Carolina, 27514, United States

    Contact

    Contact Email: •••••@•••••

  • UPMC Childrens Hospital of Pittsburgh

    AVAILABLE

    Pittsburgh, Pennsylvania, 15224, United States

    Contact

    Contact Email: •••••@•••••

  • Vanderbilt Children's Hospital

    AVAILABLE

    Nashville, Tennessee, 37232-7610, United States

    Contact

    Contact Email: •••••@•••••

  • Washington University

    AVAILABLE

    Washington, Missouri, 63130, United States

    Contact

    Contact Email: •••••@•••••